DK1916258T3 - Forøgelse af ekspression af en enkeltstrenget, heterolog nukleotidsekvens fra rekombinante, virale vektorer ved en sådan udformning af sekvensen at den danner intrastrengbasepar - Google Patents

Forøgelse af ekspression af en enkeltstrenget, heterolog nukleotidsekvens fra rekombinante, virale vektorer ved en sådan udformning af sekvensen at den danner intrastrengbasepar

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Publication number
DK1916258T3
DK1916258T3 DK07022775.6T DK07022775T DK1916258T3 DK 1916258 T3 DK1916258 T3 DK 1916258T3 DK 07022775 T DK07022775 T DK 07022775T DK 1916258 T3 DK1916258 T3 DK 1916258T3
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Denmark
Prior art keywords
sequence
base pairs
raav vector
raav
heterologous sequence
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Application number
DK07022775.6T
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English (en)
Inventor
Barrie Carter
Original Assignee
Genzyme Corp
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Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=22575432&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=DK1916258(T3) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Genzyme Corp filed Critical Genzyme Corp
Application granted granted Critical
Publication of DK1916258T3 publication Critical patent/DK1916258T3/da

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/19Cytokines; Lymphokines; Interferons
    • A61K38/191Tumor necrosis factors [TNF], e.g. lymphotoxin [LT], i.e. TNF-beta
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70578NGF-receptor/TNF-receptor superfamily, e.g. CD27, CD30, CD40, CD95
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12PFERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P21/00Preparation of peptides or proteins
    • C12P21/06Preparation of peptides or proteins produced by the hydrolysis of a peptide bond, e.g. hydrolysate products
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/30Non-immunoglobulin-derived peptide or protein having an immunoglobulin constant or Fc region, or a fragment thereof, attached thereto
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/33Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Toxicology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Cell Biology (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
DK07022775.6T 1999-08-09 2000-08-08 Forøgelse af ekspression af en enkeltstrenget, heterolog nukleotidsekvens fra rekombinante, virale vektorer ved en sådan udformning af sekvensen at den danner intrastrengbasepar DK1916258T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US16008099P 1999-08-09 1999-08-09
EP00953892A EP1204739B1 (en) 1999-08-09 2000-08-08 Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs

Publications (1)

Publication Number Publication Date
DK1916258T3 true DK1916258T3 (da) 2014-07-28

Family

ID=22575432

Family Applications (2)

Application Number Title Priority Date Filing Date
DK00953892T DK1204739T3 (da) 1999-08-09 2000-08-08 Forögelse af ekspression af en enkeltstrenget, heterolog nukleotidsekvens fra rekombinante, virale vektorer ved en sådan udformning af sekvensen at den danner intrastrengbasepar
DK07022775.6T DK1916258T3 (da) 1999-08-09 2000-08-08 Forøgelse af ekspression af en enkeltstrenget, heterolog nukleotidsekvens fra rekombinante, virale vektorer ved en sådan udformning af sekvensen at den danner intrastrengbasepar

Family Applications Before (1)

Application Number Title Priority Date Filing Date
DK00953892T DK1204739T3 (da) 1999-08-09 2000-08-08 Forögelse af ekspression af en enkeltstrenget, heterolog nukleotidsekvens fra rekombinante, virale vektorer ved en sådan udformning af sekvensen at den danner intrastrengbasepar

Country Status (12)

Country Link
US (8) US6596535B1 (da)
EP (2) EP1204739B1 (da)
JP (2) JP4827353B2 (da)
AT (1) ATE403715T1 (da)
AU (1) AU781958C (da)
CA (1) CA2379166C (da)
CY (1) CY1107432T1 (da)
DE (1) DE60039766D1 (da)
DK (2) DK1204739T3 (da)
ES (2) ES2478635T3 (da)
PT (2) PT1916258E (da)
WO (1) WO2001011034A2 (da)

Families Citing this family (312)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6924128B2 (en) 1994-12-06 2005-08-02 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant AAV vectors
US6995006B2 (en) * 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6346415B1 (en) 1997-10-21 2002-02-12 Targeted Genetics Corporation Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors
US6893865B1 (en) 1999-04-28 2005-05-17 Targeted Genetics Corporation Methods, compositions, and cells for encapsidating recombinant vectors in AAV particles
PT1916258E (pt) * 1999-08-09 2014-07-29 Genzyme Corp Aumento da expressão de uma sequência nucleotídica heteróloga de cadeia simples a partir de vectores virais recombinantes por concepção da sequência de maneira que esta forme pares de bases intracadeia
NZ522840A (en) 2000-06-01 2004-12-24 Univ North Carolina A parvovirus vector that carries a duplexed genome resulting in co-packaging of strands of plus and minus polarity tethered together
US8241622B2 (en) * 2001-07-13 2012-08-14 University Of Iowa Research Foundation Adeno-associated virus vectors with intravector heterologous terminal palindromic sequences
US20040241854A1 (en) * 2002-08-05 2004-12-02 Davidson Beverly L. siRNA-mediated gene silencing
US20040023390A1 (en) * 2002-08-05 2004-02-05 Davidson Beverly L. SiRNA-mediated gene silencing with viral vectors
US20080274989A1 (en) 2002-08-05 2008-11-06 University Of Iowa Research Foundation Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof
US20050255086A1 (en) * 2002-08-05 2005-11-17 Davidson Beverly L Nucleic acid silencing of Huntington's Disease gene
US20050042646A1 (en) * 2002-08-05 2005-02-24 Davidson Beverly L. RNA interference suppresion of neurodegenerative diseases and methods of use thereof
NZ545656A (en) 2003-09-01 2009-02-28 Amc Amsterdam AAV vectors for in vivo gene therapy of rheumatoid arthritis
EP2322638A1 (en) * 2003-09-01 2011-05-18 Academisch Medisch Centrum AAV vectors for in vivo gene therapy of rheumatoid arthritis
FR2874384B1 (fr) * 2004-08-17 2010-07-30 Genethon Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables
WO2006050211A2 (en) 2004-10-28 2006-05-11 University Of Pittsburgh Of The Commonwealth System Of Higher Education Peripherally delivered glutamic acid decarboxylase gene therapy for spinal cord injury pain
WO2007149115A1 (en) * 2006-06-15 2007-12-27 Targeted Genetics Corporation Methods for treating target joints in inflammatory arthritis using aav vectors encoding a tnf antagonist
US8192441B2 (en) * 2008-10-03 2012-06-05 Howmedica Osteonics Corp. High tibial osteotomy instrumentation
JP5879256B2 (ja) 2009-05-02 2016-03-08 ジェンザイム・コーポレーション 神経変性障害のための遺伝子治療
US20130023033A1 (en) 2010-03-29 2013-01-24 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
WO2012007458A1 (en) 2010-07-12 2012-01-19 Universidad Autónoma De Barcelona Gene therapy composition for use in diabetes treatment
ES2684307T3 (es) 2010-11-05 2018-10-02 The Board Of Trustees Of The Leland Stanford Junior University Proteínas estabilizadas de tipo opsina de función escalonada y métodos de uso de las mismas
AU2011323235B2 (en) 2010-11-05 2015-10-29 The Board Of Trustees Of The Leland Stanford Junior University Optogenetic control of reward-related behaviors
CN106376525A (zh) 2010-11-05 2017-02-08 斯坦福大学托管董事会 记忆功能的控制和表征
WO2012061679A2 (en) 2010-11-05 2012-05-10 The Board Of Trustees Of The Leland Stanford Junior University Light-activated chimeric opsins and methods of using the same
US20120263754A1 (en) 2011-02-15 2012-10-18 Immune Design Corp. Methods for Enhancing Immunogen Specific Immune Responses by Vectored Vaccines
WO2012141984A1 (en) 2011-04-08 2012-10-18 Immune Design Corp. Immunogenic compositions and methods of using the compositions for inducing humoral and cellular immune responses
WO2012142526A1 (en) 2011-04-14 2012-10-18 Modiano Jaime Use of tumor fas expression to determine response to anti-cancer therapy
WO2012145509A2 (en) 2011-04-19 2012-10-26 The Research Foundation Of State University Of New York Adeno-associated-virus rep sequences, vectors, and viruses
CA2859364C (en) 2011-12-16 2019-05-07 The Board Of Trustees Of The Leland Stanford Junior University Opsin polypeptides and methods of use thereof
EP3205374B1 (en) 2012-02-21 2019-03-27 The Board of Trustees of The Leland Stanford Junior University Compositions for treating neurogenic disorders of the pelvic floor
KR102136433B1 (ko) 2012-05-16 2020-07-22 이뮨 디자인 코포레이션 Hsv-2 백신
EP2692868A1 (en) 2012-08-02 2014-02-05 Universitat Autònoma De Barcelona Adeno-associated viral (AAV) vectors useful for transducing adipose tissue
JP2016503405A (ja) 2012-11-05 2016-02-04 ジェンザイム・コーポレーション タンパク質症を処置するための組成物および方法
JP6580487B2 (ja) 2012-11-21 2019-09-25 サーキット セラピューティクス, インコーポレイテッド 光遺伝学的治療のためのシステムおよび方法
RU2692652C2 (ru) 2013-03-13 2019-06-25 Джензим Корпорейшн Слитые белки, содержащие связывающие pdgf и vegf части, и способы их применения
SI2984166T1 (sl) 2013-03-15 2020-09-30 The Trustees Of The University Of Pennsylvania Sestavki za zdravljenje MPSI
JP6594854B2 (ja) 2013-03-15 2019-10-23 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー 行動状態の光遺伝学的制御方法
CN105682674A (zh) 2013-04-17 2016-06-15 建新公司 用于治疗和预防黄斑变性的组合物和方法
AU2014260101B2 (en) 2013-04-29 2018-07-26 Humboldt-Universitat Zu Berlin Devices, systems and methods for optogenetic modulation of action potentials in target cells
MX380973B (es) 2013-05-01 2025-03-12 Genzyme Corp Composiciones y metodos para tratar la atrofia muscular espinal.
RU2671558C2 (ru) 2013-06-14 2018-11-02 Псайоксус Терапьютикс Лимитед Режим введения и составы для аденовирусов типа в
EP3033427A4 (en) 2013-08-14 2017-05-31 The Board Of Trustees Of The University Of the Leland Stanford Junior University Compositions and methods for controlling pain
AU2014338864C1 (en) 2013-10-25 2020-07-16 Akamis Bio Limited Oncolytic adenoviruses armed with heterologous genes
TWI687225B (zh) 2014-02-06 2020-03-11 美商健臻公司 用於治療及預防黃斑部病變的組成物及方法
CA2939093A1 (en) 2014-02-14 2015-08-20 Immune Design Corp. Immunotherapy of cancer through combination of local and systemic immune stimulation
RS60902B1 (sr) 2014-03-09 2020-11-30 Univ Pennsylvania Kompozicije korisne u lečenju nedostatka ornitinske transkarbamilaze (otc)
CA2942248A1 (en) * 2014-03-20 2015-09-24 Yanmar Co., Ltd. Engine device and stationary work machine having same mounted thereon
FI3628334T3 (fi) 2014-03-21 2023-09-15 Genzyme Corp Geenihoito verkkokalvon pigmenttirappeuma
EP3122761B1 (en) 2014-03-28 2019-03-06 The Board of Trustees of The Leland Stanford Junior University Engineered light-activated anion channel proteins and methods of use thereof
WO2015164723A1 (en) 2014-04-25 2015-10-29 The Trustees Of The University Of Pennsylvania Methods and compositions for treating metastatic breast cancer and other cancers in the brain
ES2876409T3 (es) 2014-04-25 2021-11-12 Univ Pennsylvania Variantes del RLBD y su uso en composiciones para reducir los niveles de colesterol
IL248102B (en) 2014-05-02 2022-07-01 Genzyme Corp aav vectors for gene therapy of the central nervous system and retina
PE20170261A1 (es) 2014-05-13 2017-04-12 Univ Pennsylvania Composiciones que comprenden virus adenoasociado (aav) que expresa constructos de anticuerpos duales y sus usos
EP3169352A1 (en) 2014-07-15 2017-05-24 Immune Design Corp. Prime-boost regimens with a tlr4 agonist adjuvant and a lentiviral vector
EP4012035B1 (en) 2014-09-16 2024-11-06 Genzyme Corporation Adeno-associated viral vectors for treating myocilin (myoc) glaucoma
PT4012035T (pt) 2014-09-16 2025-02-11 Genzyme Corp Vetores virais adenoassociados para tratar o glaucoma de miocilina (mioc)
CA2960912A1 (en) 2014-09-16 2016-03-24 Universitat Autonoma De Barcelona Adeno-associated viral vectors for the gene therapy of metabolic diseases
EP3242945B1 (en) 2015-01-07 2021-09-01 Universitat Autònoma de Barcelona Single-vector gene construct comprising insulin and glucokinase genes
CA3301261A1 (en) 2015-01-20 2026-03-02 Genzyme Corporation Analytical ultracentrifugation for characterization of recombinant viral particles
US10676726B2 (en) 2015-02-09 2020-06-09 Duke University Compositions and methods for epigenome editing
TN2017000353A1 (en) 2015-02-10 2019-01-16 Genzyme Corp Enhanced delivery of viral particles to the striatum and cortex
TN2017000354A1 (en) 2015-02-10 2019-01-16 Genzyme Corp VARIANT RNAi
TWI707951B (zh) 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造
EP3288594B1 (en) 2015-04-27 2022-06-29 The Trustees of The University of Pennsylvania Dual aav vector system for crispr/cas9 mediated correction of human disease
EA201792500A1 (ru) 2015-05-13 2018-04-30 Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания Aav-опосредованная экспрессия антител против гриппа и способы их использования
WO2016186772A2 (en) 2015-05-16 2016-11-24 Genzyme Corporation Gene editing of deep intronic mutations
WO2017015637A1 (en) 2015-07-22 2017-01-26 Duke University High-throughput screening of regulatory element function with epigenome editing technologies
EP3332012A4 (en) 2015-08-06 2019-01-16 The Trustees of The University of Pennsylvania GLP-1 AND USE THEREOF IN COMPOSITIONS FOR THE TREATMENT OF METABOLISM DISEASES
EP3341727B1 (en) 2015-08-25 2022-08-10 Duke University Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases
HK1257024A1 (zh) 2015-08-31 2019-10-11 宾夕法尼亚大学 用於治疗伴侣动物的aav-epo
CN108291216B (zh) 2015-09-24 2022-09-16 宾夕法尼亚州大学信托人 用于治疗补体介导的疾病的组合物和方法
BR112018003665A2 (en) 2015-09-28 2018-09-25 The University Of North Carolina At Chapel Hill methods and compositions for antibody evasion viral vectors
US11273227B2 (en) 2015-10-09 2022-03-15 The Trustees Of The University Of Pennsylvania Compositions and methods useful in treating Stargardt's disease and other ocular disorders
EP4089175A1 (en) 2015-10-13 2022-11-16 Duke University Genome engineering with type i crispr systems in eukaryotic cells
HK1257801A1 (zh) 2015-10-28 2019-11-01 宾夕法尼亚州大学信托人 用於基因治疗的腺伴随病毒载体的鞘内给药
IL303850A (en) 2015-12-11 2023-08-01 Univ Pennsylvania Gene therapy for treating familial hypercholesterolemia
CA3007330A1 (en) 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Composition for treatment of crigler-najjar syndrome
US11090392B2 (en) 2015-12-14 2021-08-17 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
EA201891022A1 (ru) 2015-12-17 2019-01-31 Псайоксус Терапьютикс Лимитед Вирус, кодирующий антитело к комплексу tcr или фрагмент указанного антитела
AR108683A1 (es) * 2016-01-13 2018-09-19 Merial Inc Vectores aav recombinantes que expresan genes osteoprotectores que incluyen has2 y lubricina, de utilidad en el tratamiento de la osteoartritis y afecciones articulares relacionadas en mamíferos
EP3411488A1 (en) 2016-02-03 2018-12-12 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
PL3411484T3 (pl) 2016-02-05 2024-02-19 Emory University Wstrzykiwanie jednoniciowego lub samokomplementarnego wirusa adenosatelitarnego 9 do płynu mózgowego
WO2017139381A1 (en) 2016-02-08 2017-08-17 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
CA3198936A1 (en) 2016-03-07 2017-09-14 University Of Iowa Research Foundation Aav-mediated expression using a synthetic promoter and enhancer
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
EP3442597A1 (en) 2016-04-15 2019-02-20 The Trustees Of The University Of Pennsylvania Gene therapy for treating hemophilia b
EP4706777A2 (en) 2016-04-15 2026-03-11 Alpine Immune Sciences, Inc. Cd80 variant immunomodulatory proteins and uses thereof
IL305449B2 (en) 2016-04-15 2026-01-01 Univ Pennsylvania Gene therapy for the treatment of mucocutaneous type ii diabetes
IL262365B2 (en) 2016-04-15 2024-11-01 Alpine Immune Sciences Inc Immunomodulatory proteins ICOS ligand variants and uses thereof
JP7046828B2 (ja) 2016-04-15 2022-04-04 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 滲出型加齢性黄斑変性の治療のための組成物
US11401527B2 (en) 2016-04-17 2022-08-02 The Trustees Of The University Of Pennsylvania Compositions and methods useful for prophylaxis of organophosphates
US12173305B2 (en) 2016-05-26 2024-12-24 University Of Iowa Research Foundation cis and trans requirements for terminal resolution of human bocavirus 1
US11197936B2 (en) 2016-07-08 2021-12-14 The Trustees Of The University Of Pennsylvania Methods and compositions for treatment of disorders and diseases involving RDH12
WO2018017754A1 (en) 2016-07-19 2018-01-25 Duke University Therapeutic applications of cpf1-based genome editing
WO2018022511A1 (en) 2016-07-25 2018-02-01 The Trustees Of The University Of Pennsylvania Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof
US11834490B2 (en) 2016-07-28 2023-12-05 Alpine Immune Sciences, Inc. CD112 variant immunomodulatory proteins and uses thereof
CN110088127A (zh) 2016-07-28 2019-08-02 高山免疫科学股份有限公司 Cd155变体免疫调节蛋白及其用途
US11471488B2 (en) 2016-07-28 2022-10-18 Alpine Immune Sciences, Inc. CD155 variant immunomodulatory proteins and uses thereof
NZ791267A (en) 2016-08-15 2025-11-28 Genzyme Corp Methods for detecting AAV
GB201713765D0 (en) 2017-08-28 2017-10-11 Psioxus Therapeutics Ltd Modified adenovirus
CN119614520A (zh) 2016-08-29 2025-03-14 阿卡米斯生物公司 携带双特异性t细胞衔接器的腺病毒
US11142775B2 (en) 2017-01-13 2021-10-12 University Of Iowa Research Foundation Bocaparvovirus small noncoding RNA and uses thereof
CA3049915A1 (en) 2017-01-31 2018-08-09 Stephen YOO Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua)
US11554147B2 (en) 2017-02-20 2023-01-17 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
CA3052487A1 (en) 2017-02-28 2018-09-07 The Trustees Of The University Of Pennsylvania Influenza vaccines based on aav vectors
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
JP7455579B2 (ja) 2017-02-28 2024-03-26 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 新規アデノ随伴ウイルス(aav)クレードfベクター及びその用途
BR112019017327A2 (pt) 2017-03-01 2020-04-14 Univ Pennsylvania terapia gênica para distúrbios oculares
JP2020511144A (ja) 2017-03-16 2020-04-16 アルパイン イミューン サイエンシズ インコーポレイテッド Pd−l2バリアント免疫調節タンパク質及びその使用
EP3596114A2 (en) 2017-03-16 2020-01-22 Alpine Immune Sciences, Inc. Cd80 variant immunomodulatory proteins and uses thereof
PT3596116T (pt) 2017-03-16 2023-12-04 Alpine Immune Sciences Inc Proteínas imunomoduladoras de pd-l1 variante e suas utilizações
MX2019011599A (es) 2017-03-30 2019-12-19 Univ Queensland Moleculas quimericas y usos de las mismas.
JP2020516291A (ja) 2017-04-14 2020-06-11 レジェンクスバイオ インコーポレーテッド ヒト神経細胞又はグリア細胞により生産された組換えヒトイズロン酸−2−スルファターゼ(ids)によるムコ多糖症ii型の治療
WO2018200542A1 (en) 2017-04-24 2018-11-01 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
KR102719222B1 (ko) 2017-05-11 2024-10-21 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 신경 세로이드 지질갈색소증에 대한 유전자 요법
IL306098A (en) 2017-05-24 2023-11-01 Univ Barcelona Autonoma Viral expression vectors containing the coding sequence for FIBROBLAST GROWTH FACTOR 21 (FGF21)
EP3630986B1 (en) 2017-05-31 2026-03-25 The Trustees of The University of Pennsylvania Gene therapy for treating peroxisomal disorders
PL3630143T3 (pl) * 2017-06-01 2023-10-30 Akamis Bio Limited Wirus onkolityczny i sposób
US11827898B2 (en) 2017-06-14 2023-11-28 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
MA50016A (fr) 2017-07-06 2020-07-08 Univ Pennsylvania Thérapie génique médiée par aav9 pour traiter la mucopolysaccharidose de type i
WO2019040994A1 (en) 2017-09-01 2019-03-07 The Australian National University IMMUNOREGULATOR MOLECULES AND USES THEREOF
TWI835747B (zh) 2017-09-22 2024-03-21 賓州大學委員會 用於治療黏多醣病 ii 型之基因治療
KR102686857B1 (ko) 2017-09-22 2024-07-18 젠자임 코포레이션 변이체 RNAi
EP3687565A1 (en) 2017-09-27 2020-08-05 Alexion Pharmaceuticals, Inc. Methods of improving cardiovascular function and treating cardiovascular disease using a recombinant ectonucleotide pyrophosphatase phosphodiesterase (npp1)
KR102813968B1 (ko) 2017-10-10 2025-05-29 알파인 이뮨 사이언시즈, 인코포레이티드 Ctla-4 변이체 면역조절 단백질 및 이의 용도
BR112020007405A2 (pt) 2017-10-18 2020-12-08 Regenxbio Inc. Produtos terapêuticos de anticorpo pós-translacionalmente modificados totalmente humanos
AU2018350990A1 (en) 2017-10-18 2020-05-21 Regenxbio Inc. Treatment of ocular diseases and metastatic colon cancer with human post-translationally modified VEGF-Trap
SMT202600062T1 (it) 2017-10-18 2026-03-09 Alpine Immune Sciences Inc Proteine immunomodulatrici del ligando di icos variante e composizioni e metodi correlati
KR20200104864A (ko) 2017-11-30 2020-09-04 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 뮤코다당류증 iiib형에 대한 유전자 요법
KR102823833B1 (ko) 2017-11-30 2025-06-23 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 뮤코다당류증 iiia형에 대한 유전자 요법
SG11202006148UA (en) 2018-01-03 2020-07-29 Alpine Immune Sciences Inc Multi-domain immunomodulatory proteins and methods of use thereof
GB201801614D0 (en) 2018-01-31 2018-03-14 Psioxus Therapeutics Ltd Formulation
US12558434B2 (en) 2018-02-20 2026-02-24 The Trustees Of The University Of Pennsylvania Compositions for treatment of wet age-related macular degeneration
MX2020008932A (es) 2018-02-27 2020-10-01 Univ Pennsylvania Novedosos vectores de virus adenoasociado (aav), vectores de aav que tienen una desamidacion reducida de la capside y usos de estos.
US20210363191A1 (en) 2018-04-03 2021-11-25 Stridebio, Inc. Antibody-evading virus vectors
CA3094465A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
CN112543766A (zh) 2018-04-03 2021-03-23 斯特里迪比奥公司 抗体逃避性病毒载体
US12460226B2 (en) 2018-04-16 2025-11-04 The Trustees Of The University Of Pennsylvania Compositions and methods for treating duchenne muscular dystrophy
WO2019212922A1 (en) 2018-04-29 2019-11-07 Regenxbio Inc. Systems and methods of spectrophotometry for the determination of genome content, capsid content and full/empty ratios of adeno-associated virus particles
WO2019212921A1 (en) 2018-04-29 2019-11-07 Regenxbio Inc. Scalable clarification process for recombinant aav production
WO2019241535A2 (en) 2018-06-14 2019-12-19 Regenxbio Inc. Anion exchange chromatography for recombinant aav production
US12065476B2 (en) 2018-06-15 2024-08-20 Alpine Immune Sciences, Inc. PD-1 variant immunomodulatory proteins and uses thereof
EP3830265A1 (en) 2018-08-03 2021-06-09 Genzyme Corporation Variant rnai against alpha-synuclein
EP3833745A1 (en) 2018-08-10 2021-06-16 REGENXBIO Inc. Scalable method for recombinant aav production
CN110857440B (zh) 2018-08-23 2021-02-19 武汉纽福斯生物科技有限公司 重组人ⅱ型线粒体动力蛋白样gtp酶基因序列及其应用
AU2019345151B2 (en) 2018-09-19 2025-04-17 Alpine Immune Sciences, Inc. Methods and uses of variant CD80 fusion proteins and related constructs
EP4461814A3 (en) 2018-10-12 2025-02-26 Genzyme Corporation Generation of improved human pah for treatment of severe pku by liver-directed gene replacement therapy
UY38407A (es) 2018-10-15 2020-05-29 Novartis Ag Anticuerpos estabilizadores de trem2
JP2022514112A (ja) 2018-10-15 2022-02-09 リジェネクスバイオ インコーポレイテッド 複製欠損型ウイルスベクター及びウイルスの感染性を測定するための方法
CN111118016B (zh) 2018-10-30 2022-04-15 上海朗昇生物科技有限公司 治疗视网膜色素变性疾病的基因治疗载体
US12370240B2 (en) 2018-11-28 2025-07-29 Genascence Corporation Methods and compositions for treating osteoarthritis
WO2020113141A2 (en) 2018-11-30 2020-06-04 Alpine Immune Sciences, Inc. Cd86 variant immunomodulatory proteins and uses thereof
WO2020142714A1 (en) 2019-01-04 2020-07-09 Exonics Therapeutics, Inc. Aav expression cassette and aav vectors comprising the same
TW202045728A (zh) 2019-02-26 2020-12-16 賓州大學委員會 用於治療克拉培氏病之組成物
EP3934699A4 (en) 2019-03-04 2022-12-21 The Trustees of The University of Pennsylvania NEUROPROTECTIVE GENE THERAPY TARGETING THE AKT PATHWAY
CN111718418B (zh) 2019-03-19 2021-08-27 华东师范大学 一种增强基因编辑的融合蛋白及其应用
BR112021018776A2 (pt) 2019-03-21 2021-11-30 Stridebio Inc Vetores de vírus adenoassociado recombinante
CN114450411A (zh) 2019-04-01 2022-05-06 特纳亚治疗股份有限公司 具有工程化衣壳的腺相关病毒
EP4667578A3 (en) 2019-04-03 2026-04-01 REGENXBIO Inc. Gene therapy for eye pathologies
TW202102526A (zh) 2019-04-04 2021-01-16 美商銳進科斯生物股份有限公司 重組腺相關病毒及其用途
HUE064411T2 (hu) 2019-04-11 2024-03-28 Regenxbio Inc Méretkizárásos kromatográfiás módszerek rekombináns adeno-asszociált víruskészítmények jellemzésére
EP4438054A3 (en) 2019-04-17 2025-01-08 Alpine Immune Sciences, Inc. Methods and uses of variant icos ligand (icosl) fusion proteins
US20220143115A1 (en) 2019-04-19 2022-05-12 Regenxbio Inc. Adeno-Associated Virus Vector Formulations and Methods
KR20220012231A (ko) 2019-04-24 2022-02-03 리젠엑스바이오 인크. 완전-인간 번역 후 변형된 항체 치료제
BR112021021908A2 (pt) 2019-05-03 2022-02-01 Univ Pennsylvania Composições úteis no tratamento da leucodistrofia metacromática
CN112180094A (zh) 2019-07-04 2021-01-05 上海东慈生物科技有限公司 Dkk1抑制剂在预防和/或治疗肿瘤恶病质与糖尿病伴随疾病中的应用
US20220251567A1 (en) 2019-07-10 2022-08-11 Inserm (Institut National De La Santè Et De La Recherche Médicale) Methods for the treatment of epilepsy
EP3914720B9 (en) 2019-07-10 2023-09-27 Masonic Medical Research Laboratory Vgll4 with ucp-1 cis-regulatory element and method of use thereof
JP2022544004A (ja) 2019-07-26 2022-10-17 リジェネックスバイオ インコーポレイテッド 操作された核酸調節エレメントならびにその使用方法
JP2022543722A (ja) 2019-08-16 2022-10-14 センター フォー エクセレンス イン ブレイン サイエンス アンド インテリジェンス テクノロジー,チャイニーズ アカデミー オブ サイエンシーズ 機能的ニューロン死に関連する神経系疾患の予防および/または治療におけるPtbp1阻害剤の使用
WO2021041373A1 (en) 2019-08-26 2021-03-04 Regenxbio Inc. Treatment of diabetic retinopathy with fully-human post-translationally modified anti-vegf fab
AU2020362119A1 (en) 2019-10-07 2022-05-26 Regenxbio Inc. Adeno-associated virus vector pharmaceutical composition and methods
WO2021076600A1 (en) 2019-10-14 2021-04-22 Duke University Compositions comprising novel prokaryotic sodium channels and associated methods
KR20220083714A (ko) 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터
US20230016983A1 (en) 2019-11-19 2023-01-19 lNSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) Antisense oligonucleotides and thier use for the treatment of cancer
MX2022006427A (es) 2019-11-28 2022-09-07 Regenxbio Inc "construcciones para terapia génica con microdistrofina y uso de las mismas.
WO2021150570A1 (en) 2020-01-22 2021-07-29 Regenxbio Inc. Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua)
CA3167741A1 (en) 2020-01-29 2021-08-05 Regenxbio Inc. Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids) produced by human neural or glial cells
AR121201A1 (es) 2020-01-29 2022-04-27 Regenxbio Inc Tratamiento de mucopolisacaridosis iva
GB202004498D0 (en) 2020-03-27 2020-05-13 Ucl Business Ltd Activity-dependent gene therapy for neurological disorders
JP2023528139A (ja) 2020-03-30 2023-07-04 ウーハン ニューロフス バイオテクノロジー リミテッド カンパニー ヒト核因子e2関連因子2の発現ベクター及び発現ベクターの適用
JP7748393B2 (ja) 2020-05-08 2025-10-02 アルパイン イミューン サイエンシズ インコーポレイテッド Aprilおよびbaff阻害免疫調節タンパク質、ならびにその使用方法
CA3177924A1 (en) 2020-05-12 2021-11-18 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of krabbe disease
US20230304034A1 (en) 2020-05-12 2023-09-28 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
US20230220069A1 (en) 2020-06-17 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of gene therapy patients
KR20230050336A (ko) 2020-07-10 2023-04-14 인스티튜트 내셔널 드 라 싼테 에 드 라 리셰르셰 메디칼르 (인 썸) 뇌전증을 치료하기 위한 방법과 조성물
GB202010981D0 (en) 2020-07-16 2020-09-02 Ucl Business Ltd Gene therapy for neuromuscular and neuromotor disorders
US20230265456A1 (en) 2020-08-10 2023-08-24 Fundacion Para La Investigacion Medica Aplicada Gene therapy vector expressing cyp27a1 for the treatment of cerebrotendinous xanthomatosis
EP4200408A1 (en) 2020-08-19 2023-06-28 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of rett syndrome
BR112023003310A2 (pt) 2020-08-24 2023-05-02 Univ Pennsylvania Vetores virais que codificam fusões de agonistas do receptor de glp-1 e usos dos mesmos no tratamento de doenças metabólicas
EP4213890A1 (en) 2020-09-15 2023-07-26 RegenxBio Inc. Vectorized lanadelumab and administration thereof
WO2022060916A1 (en) 2020-09-15 2022-03-24 Regenxbio Inc. Vectorized antibodies for anti-viral therapy
JP2023544165A (ja) 2020-10-01 2023-10-20 ジェンザイム・コーポレーション 肝臓に向けられた遺伝子補充療法によってpkuを処置するためのヒトpah発現カセット
US20240024508A1 (en) 2020-10-07 2024-01-25 Regenxbio Inc. Formulations for suprachoroidal administration such as high viscosity formulations
US20230372538A1 (en) 2020-10-07 2023-11-23 Regenxbio Inc. Formulations for suprachoroidal administration such as formulations with aggregate formation
US20230381341A1 (en) 2020-10-07 2023-11-30 Regenxbio Inc. Adeno-associated viruses for ocular delivery of gene therapy
WO2022076750A2 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Recombinant adeno-associated viruses for cns or muscle delivery
CA3197342A1 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Gene therapy for ocular manifestations of cln2 disease
CA3194861A1 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Formulations for suprachoroidal administration such as gel formulations
AU2021356684A1 (en) 2020-10-09 2023-05-11 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
IL301675A (en) 2020-10-09 2023-05-01 Tenaya Therapeutics Inc Methods and preparations for placophilin-2 gene therapy
IL302127A (en) 2020-10-18 2023-06-01 Univ Pennsylvania An improved adeno-associated virus (AAV) vector and its uses
CN116528892A (zh) 2020-10-28 2023-08-01 再生生物股份有限公司 用于眼部适应症的载体化抗TNF-α抗体
WO2022094157A1 (en) 2020-10-28 2022-05-05 Regenxbio Inc. Vectorized anti-cgrp and anti-cgrpr antibodies and administration thereof
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
CN116457373A (zh) 2020-10-29 2023-07-18 再生生物股份有限公司 用于眼部适应症的载体化TNF-α拮抗剂
US20230390418A1 (en) 2020-10-29 2023-12-07 Regenxbio Inc. Vectorized factor xii antibodies and administration thereof
AU2021369793A1 (en) 2020-10-29 2023-06-08 The Trustees Of The University Of Pennsylvania Aav capsids and compositions containing same
JP2024016295A (ja) * 2020-11-30 2024-02-07 タカラバイオ株式会社 二価の陽イオンを利用した非エンベロープウイルスの製造方法
TW202237850A (zh) 2020-12-01 2022-10-01 賓州大學委員會 具有組織特異性靶向基序的新穎構成物及含有其之組成物
EP4263841A1 (en) 2020-12-16 2023-10-25 REGENXBIO Inc. Method of producing a recombinant adeno-associated virus particle
US20240091382A1 (en) 2020-12-23 2024-03-21 Vivet Therapeutics Minimal bile acid inducible promoters for gene therapy
WO2022147087A1 (en) 2020-12-29 2022-07-07 Regenxbio Inc. Tau-specific antibody gene therapy compositions, methods and uses thereof
EP4281568A1 (en) 2021-01-21 2023-11-29 RegenxBio Inc. Improved production of recombinant polypeptides and viruses
WO2022165313A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
BR112023016075A2 (pt) 2021-02-10 2023-11-21 Regenxbio Inc Método para tratar hepatoesplenomegalia, método de distribuição de um raav que codifica hids, método para tratar um sujeito humano, método de tratamento de um sujeito humano, método para determinar a eficácia do tratamento, método para identificar um sujeito como tendo mps ii
CN117460832A (zh) 2021-03-22 2024-01-26 建新公司 空aav衣壳和完整aav衣壳的尺寸排阻色谱分析
AR125406A1 (es) 2021-04-23 2023-07-12 Univ Pennsylvania Nuevas composiciones con motivos selectivos para el cerebro y composiciones que las contienen
KR20240004564A (ko) 2021-04-26 2024-01-11 리젠엑스바이오 인크. 디스트로핀병증의 치료를 위한 마이크로디스트로핀 유전자 치료법 투여
WO2022235614A2 (en) 2021-05-04 2022-11-10 Regenxbio Inc. Novel aav vectors and methods and uses thereof
KR20240019124A (ko) 2021-05-07 2024-02-14 알파인 이뮨 사이언시즈, 인코포레이티드 Taci-fc 융합 면역조절 단백질의 투여 및 치료 방법
WO2022241030A1 (en) 2021-05-11 2022-11-17 Regenxbio Inc. Treatment of duchenne muscular dystrophy and combinations thereof
AU2022313258A1 (en) 2021-07-19 2024-02-08 New York University Auf1 combination therapies for treatment of muscle degenerative disease
CA3226402A1 (en) 2021-07-19 2023-01-26 Dounia ABBADI Adeno-associated viral vector compositions and methods of promoting muscle regeneration
WO2023010135A1 (en) 2021-07-30 2023-02-02 Tune Therapeutics, Inc. Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2)
EP4377459A2 (en) 2021-07-30 2024-06-05 Tune Therapeutics, Inc. Compositions and methods for modulating expression of frataxin (fxn)
EP4409010A1 (en) 2021-10-02 2024-08-07 The Trustees of The University of Pennsylvania Novel aav capsids and compositions containing same
WO2023060113A1 (en) 2021-10-05 2023-04-13 Regenxbio Inc. Compositions and methods for recombinant aav production
CN118202060A (zh) 2021-10-05 2024-06-14 再生生物股份有限公司 用于重组aav生产的组合物和方法
WO2023060272A2 (en) 2021-10-07 2023-04-13 Regenxbio Inc. Recombinant adeno-associated viruses for cns tropic delivery
WO2023060269A1 (en) 2021-10-07 2023-04-13 Regenxbio Inc. Recombinant adeno-associated viruses for targeted delivery
US20240404630A1 (en) * 2021-10-13 2024-12-05 Haystackanalyt Ics Pvt. Ltd. Systems and methods for secure genomic analysis using a specialized edge computing device
EP4423285A1 (en) 2021-10-28 2024-09-04 RegenxBio Inc. Engineered nucleic acid regulatory elements and methods and uses thereof
WO2023087019A2 (en) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
WO2023102517A1 (en) 2021-12-02 2023-06-08 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
WO2023131682A1 (en) 2022-01-06 2023-07-13 Ucl Business Ltd Endogenous gene regulation to treat neurological disorders and diseases
US20250177495A1 (en) 2022-01-10 2025-06-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of metachromatic leukodystrophy
US20250144243A1 (en) 2022-01-25 2025-05-08 The Trustees Of The University Of Pennsylvania Aav capsids for improved heart transduction and detargeting of liver
GB202201744D0 (en) 2022-02-10 2022-03-30 Ucl Business Ltd Treatment of acquired focal epilepsy
WO2023172883A1 (en) 2022-03-07 2023-09-14 Alpine Immune Sciences, Inc. Immunomodulatory proteins of variant cd80 polypeptides, cell therapies thereof and related methods and uses
EP4493226A1 (en) 2022-03-13 2025-01-22 RegenxBio Inc. Modified muscle-specific promoters
WO2023183623A1 (en) 2022-03-25 2023-09-28 Regenxbio Inc. Dominant-negative tumor necrosis factor alpha adeno-associated virus gene therapy
JP2025512969A (ja) 2022-04-06 2025-04-22 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Her2陽性転移性乳がん及び他のがんを治療するための組成物及び方法
CA3247417A1 (en) 2022-04-06 2023-10-12 Genzyme Corporation TARGETED GENE THERAPY FOR DM-1 MYOTONIC DYSTROPHY
US20250250326A1 (en) 2022-04-06 2025-08-07 The Trustees Of The University Of Pennsylvania Passive immunization with anti-aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
TW202404651A (zh) 2022-04-06 2024-02-01 美商銳進科斯生物股份有限公司 用於脈絡膜上投與之調配物諸如形成聚集體之調配物
CA3247507A1 (en) 2022-04-06 2023-10-12 Regenxbio Inc. PHARMACEUTICAL COMPOSITION COMPRISING A RECOMMENDED ADENO-ASSOCIATED VIRUS (AAV) VECTOR WITH AN EXPRESSION CASSETTE ENCODED BY A TRANSGENE FOR SUPRACHOROID ADMINISTRATION
TW202345913A (zh) 2022-04-06 2023-12-01 美商銳進科斯生物股份有限公司 用於脈絡膜上投與之調配物諸如凝膠調配物
JP2025512333A (ja) 2022-04-11 2025-04-17 テナヤ セラピューティクス, インコーポレイテッド 操作されたカプシドを伴うアデノ随伴ウイルス
CN119630802A (zh) 2022-04-12 2025-03-14 建新公司 Irak4调节剂用于基因疗法的用途
WO2023201274A1 (en) 2022-04-12 2023-10-19 Genzyme Corporation Use of an irak4 modulator for gene therapy
IL316213A (en) 2022-04-12 2024-12-01 Genzyme Corp Dendritic cell testing for innate immunogenicity to gene therapy agents
WO2023201277A1 (en) 2022-04-14 2023-10-19 Regenxbio Inc. Recombinant adeno-associated viruses for cns tropic delivery
WO2023201308A1 (en) 2022-04-14 2023-10-19 Regenxbio Inc. Gene therapy for treating an ocular disease
US20250277004A1 (en) 2022-04-18 2025-09-04 Regenxbio Inc. Hybrid aav capsids
EP4518972A2 (en) 2022-05-03 2025-03-12 RegenxBio Inc. Vectorized anti-complement antibodies and complement agents and administration thereof
US20250288697A1 (en) 2022-05-03 2025-09-18 Regenxbio Inc. Vectorized anti-tnf-alpha inhibitors for ocular indications
US20250353883A1 (en) 2022-05-06 2025-11-20 Novartis Ag Novel recombinant aav vp2 fusion polypeptides
AU2023273809A1 (en) 2022-05-16 2025-01-02 Genzyme Corporation Methods of treating metachromatic leukodystrophy
CA3251359A1 (en) 2022-05-17 2023-11-23 École Polytechnique Fédérale de Lausanne BIOSENSORS DESIGNED FOR ENHANCED T LYMPHOCYTE THERAPY
WO2023239627A2 (en) 2022-06-08 2023-12-14 Regenxbio Inc. Methods for recombinant aav production
IL317874A (en) 2022-06-24 2025-02-01 Tune Therapeutics Inc Compounds, systems and methods for reducing low density lipoproteins through targeted gene suppression
CA3261865A1 (en) 2022-07-12 2024-01-18 Tune Therapeutics, Inc. TARGETED TRANSCRIPTIONAL ACTIVATION COMPOSITIONS, SYSTEMS AND METHODS
WO2024015966A2 (en) 2022-07-15 2024-01-18 The Trustees Of The University Of Pennsylvania Recombinant aav having aav clade d and clade e capsids and compositions containing same
EP4558149A1 (en) 2022-07-21 2025-05-28 Institut National de la Santé et de la Recherche Médicale Methods and compositions for treating chronic pain disorders
JP2025527658A (ja) 2022-08-24 2025-08-22 リジェネックスバイオ インコーポレイテッド 組換えアデノ随伴ウイルス及びその使用
IL319873A (en) 2022-09-30 2025-05-01 Regenxbio Inc Treatment of eye diseases with recombinant viral vectors containing anti-VEGF FAB
WO2024077018A2 (en) 2022-10-04 2024-04-11 Alpine Immune Sciences, Inc. Methods and uses of taci-fc fusion immunomodulatory protein
JP2025534666A (ja) 2022-10-11 2025-10-17 リジェネックスバイオ インコーポレイテッド 操作された核酸調節エレメントならびにその使用方法
CN117947040A (zh) 2022-10-31 2024-04-30 苏州荷光科汇生物科技有限公司 用于目的基因的表达盒及其应用
KR20250135916A (ko) 2022-12-17 2025-09-15 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 심장 및 골격근 특이적 표적화 모티프를 갖는 재조합 aav 돌연변이체 벡터 및 이를 포함하는 조성물
WO2024130070A2 (en) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof
EP4646478A1 (en) 2023-01-06 2025-11-12 Institut National de la Santé et de la Recherche Médicale Intravenous administration of antisense oligonucleotides for the treatment of pain
WO2024163683A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Systems, compositions, and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) and x-inactive specific transcript (xist)
WO2024163678A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods
CN118576695A (zh) 2023-03-01 2024-09-03 上海三弹生物医药科技有限公司 一种治疗或改善恶病质的方法
EP4680741A2 (en) 2023-03-15 2026-01-21 RegenxBio Inc. Exon skipping gene therapy constructs, vectors and uses thereof
JP2026511976A (ja) 2023-04-05 2026-04-14 ジェンザイム・コーポレーション Dm-1筋強直性ジストロフィーの標的遺伝子療法
WO2024211780A1 (en) 2023-04-07 2024-10-10 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2024215653A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Guide rnas, vectors, and virions for targeting mutations in the pln gene
WO2024215655A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Cardioprotective bag3 therapies
EP4694904A2 (en) 2023-04-13 2026-02-18 REGENXBIO Inc. Targeting aav capsids, methods of manufacturing and using same
EP4709866A2 (en) 2023-05-07 2026-03-18 REGENXBIO Inc. Compositions and methods for recombinant aav production
WO2024238853A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Adeno-associated viruses for ocular delivery of gene therapy
WO2024238859A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Vectorized c5 inhibitor agents and administration thereof
AU2024271004A1 (en) 2023-05-16 2026-01-15 Regenxbio Inc. Vectorized anti-complement antibodies and administration thereof
EP4724085A1 (en) 2023-06-12 2026-04-15 The Trustees of The University of Pennsylvania Aav gene therapy for mucopolysaccharidosis iiib
AR133098A1 (es) 2023-06-29 2025-08-27 Univ Pennsylvania Aav mutante con motivos dirigidos al sistema nervioso central y composiciones que lo contienen
WO2025008406A1 (en) 2023-07-04 2025-01-09 Institut National de la Santé et de la Recherche Médicale Antisense oligonucleotides and their use for the treatment of cancer
CN116790615B (zh) 2023-07-11 2024-11-22 康霖生物科技(杭州)有限公司 一种用于过敏性疾病的基因治疗载体核酸构建体及其使用方法
WO2025016362A1 (zh) 2023-07-19 2025-01-23 中国科学院脑科学与智能技术卓越创新中心 基于脑内磷稳态促进剂的疗法
IL326390A (en) 2023-08-10 2026-04-01 Univ Pennsylvania Compositions and methods for treating spinal muscular atrophy
WO2025075963A1 (en) 2023-10-02 2025-04-10 Regenxbio Inc. Methods and formulations for treating mucopolysaccharidosis ii-associated hearing loss with recombinant human iduronate-2-sulfatase
WO2025090962A1 (en) 2023-10-25 2025-05-01 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2025102034A1 (en) 2023-11-10 2025-05-15 The Trustees Of The University Of Pennsylvania Gene therapy for barth syndrome
WO2025106661A1 (en) 2023-11-14 2025-05-22 The Trustees Of The University Of Pennsylvania Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof
US20250152747A1 (en) 2023-11-15 2025-05-15 Genzyme Corporation Methods of treating gaucher disease and gba-parkinson’s disease
WO2025106874A1 (en) 2023-11-15 2025-05-22 Genzyme Corporation Methods of treating neurodegenerative disorders
WO2025128817A1 (en) 2023-12-15 2025-06-19 Genzyme Corporation Artificial micrornas targeting tau
WO2025129157A1 (en) 2023-12-15 2025-06-19 The Trustees Of The University Of Pennsylvania Gene therapy for treatment of canavan disease
WO2025158385A1 (en) 2024-01-25 2025-07-31 Genzyme Corporation Pegylated il-2 for suppressing adaptive immune response to gene therapy
WO2025160452A1 (en) 2024-01-25 2025-07-31 Genzyme Corporation Methods for fus-based delivery of viral particles to the brain
TW202548014A (zh) 2024-01-26 2025-12-16 美商健臻公司 靶向亨丁頓舞蹈症之人工microrna
WO2025160429A1 (en) 2024-01-26 2025-07-31 Genzyme Corporation Artificial micrornas targeting snca
WO2025189030A1 (en) 2024-03-07 2025-09-12 Stellaromics, Inc. Methods and systems for volumetric imaging
WO2025217214A2 (en) 2024-04-08 2025-10-16 Regenxbio Inc. Recombinant adeno-associated viruses and uses thereof
WO2025217230A1 (en) 2024-04-08 2025-10-16 Regenxbio Inc. Vectorized anti-complement antibodies and complement agents and administration thereof
WO2025235643A1 (en) 2024-05-08 2025-11-13 Genzyme Corporation Profiling of gene therapy agents
WO2025237990A1 (en) 2024-05-14 2025-11-20 Institut National de la Santé et de la Recherche Médicale Antisense oligonucleotides and their use for the treatment of pulmonary fibrosis
WO2026006341A1 (en) 2024-06-24 2026-01-02 Regenxbio Inc. Microdystrophin gene therapy administration for treatment of dystrophinopathies

Family Cites Families (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5399346A (en) 1989-06-14 1995-03-21 The United States Of America As Represented By The Department Of Health And Human Services Gene therapy
US5605690A (en) 1989-09-05 1997-02-25 Immunex Corporation Methods of lowering active TNF-α levels in mammals using tumor necrosis factor receptor
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US5869305A (en) * 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
US6686200B1 (en) * 1993-08-31 2004-02-03 Uab Research Foundation Methods and compositions for the large scale production of recombinant adeno-associated virus
PT733103E (pt) 1993-11-09 2004-07-30 Targeted Genetics Corp Criacao de elevados titulos de vectores de aav recombinantes
PT728214E (pt) 1993-11-09 2004-11-30 Ohio Med College Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
AU707866B2 (en) 1994-12-06 1999-07-22 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant AAV vectors
US5756283A (en) 1995-06-05 1998-05-26 The Trustees Of The University Of Pennsylvania Method for improved production of recombinant adeno-associated viruses for gene therapy
US6281010B1 (en) 1995-06-05 2001-08-28 The Trustees Of The University Of Pennsylvania Adenovirus gene therapy vehicle and cell line
US5677158A (en) * 1995-06-07 1997-10-14 Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US6004797A (en) 1995-11-09 1999-12-21 Avigen, Inc. Adenovirus helper-free recombinant AAV Virion production
WO1997032990A1 (en) 1996-03-04 1997-09-12 Targeted Genetics Corporation Methods for transducing cells in blood vessels using recombinant aav vectors
AU741605B2 (en) 1996-12-18 2001-12-06 Targeted Genetics Corporation AAV split-packaging genes and cell lines comprising such genes for use in the production of recombinant AAV vectors
CA2269661A1 (en) 1996-12-18 1998-06-25 Targeted Genetics Corporation Recombinase-activatable aav packaging cassettes for use in the production of aav vectors
CA2278734A1 (en) 1997-01-23 1998-07-30 Immusol Incorporated Gene functional analysis and discovery using randomized or target-specific ribozyme gene vector libraries
US6156303A (en) * 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
JP2001514845A (ja) 1997-09-05 2001-09-18 ターゲティッド ジェネティクス コーポレイション 組換えaavベクターの高力価ヘルパーなし調製物を生成するための方法
DE69840948D1 (de) 1997-10-21 2009-08-13 Targeted Genetics Corp Invertierte terminale repeats von aav, die für die transkription aktiviert sind, zur verwendung in rekombinanten aav-vektoren
WO1999020779A1 (en) 1997-10-21 1999-04-29 Targeted Genetics Corporation Amplifiable adeno-associated virus (aav) packaging cassettes for the production of recombinant aav vectors
ES2317709T3 (es) 1998-09-04 2009-04-16 Targeted Genetics Corporation Metodos para generar preparaciones libres de auxiliares de titulo alto de vectores de aav recombinantes liberados.
US6893865B1 (en) 1999-04-28 2005-05-17 Targeted Genetics Corporation Methods, compositions, and cells for encapsidating recombinant vectors in AAV particles
DE60040147D1 (de) 1999-05-28 2008-10-16 Targeted Genetics Corp Methoden und zusammensetzungen zur reduktion des tumor-nekrosis-faktor-spiegels (tnf) bei tnf-assoziierten erkrankungen
PT1916258E (pt) 1999-08-09 2014-07-29 Genzyme Corp Aumento da expressão de uma sequência nucleotídica heteróloga de cadeia simples a partir de vectores virais recombinantes por concepção da sequência de maneira que esta forme pares de bases intracadeia
NZ522840A (en) * 2000-06-01 2004-12-24 Univ North Carolina A parvovirus vector that carries a duplexed genome resulting in co-packaging of strands of plus and minus polarity tethered together
JP6143466B2 (ja) 2013-01-10 2017-06-07 株式会社ジャパンディスプレイ タッチパネル内蔵型液晶表示装置

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